jefferies healthcare conference inc.pdf4 international demand accounts for 90% of global ivf *donor...
TRANSCRIPT
2
Forward-Looking Statements
Various statements we make in this presentation concerning our future expectations, plans and prospects, including, without limitation, statements related to our strategy, commercialization and development plans and our treatment and business goals, including statements relating to our plans to launch the AUGMENTSM
treatment in new regions, our AUGMENT treatment cycle goals and our pricing plans, our optimization and launch goals for the OvaPrimeSM treatment, our development plans for the OvaTureSM treatment, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including risks related to: the possibility that international IVF clinics may decide not to begin or continue providing the AUGMENT treatment; our expectation that the AUGMENT treatment and OvaPrime treatment meet the requirements of a class of products exempt from premarket review and approval under applicable regulations in those countries where we have launched or plan to launch; the commercial ramp up of the AUGMENT treatment, which we expect will depend upon the successful transition of ACE clinics to commercial operations, the addition of new ACE clinics, and the results from ACE clinic experience as they become available; the science underlying our treatments, which is unproven; our ability to obtain, maintain and protect intellectual property; our ability to obtain additional funding to support our business activities; our dependence on third parties for development, manufacture, marketing, sales and distribution of products; the successful commercialization and development of our treatments on the timelines we expect, if at all; obtaining necessary regulatory approvals for our potential treatments; competition from others developing treatments for similar uses; and our short operating history; as well as those risks more fully discussed in the “Risk Factors” section of our most recent 10-K and 10-Q and other reports on file with the Securities and Exchange Commission. In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. We do not assume any obligation to update any forward-looking statement.
3
OvaScience: Building a Leading Fertility Company
Fertility Treatment is a Large and Growing Market
1 in 6 couples worldwide diagnosed with infertility
90% of market is international
Mainly self-pay
AUGMENTSM treatment launched
OvaPrimeSM treatment introduction planned for end of 2015
OvaTureSM treatment in preclinical development
Proven Leadership
Proprietary Platform
The AUGMENT treatment is not available in the United States
New Fertility Treatments
4
International Demand Accounts for 90% of Global IVF
*Donor egg restrictions in these regionsMiddle East includes: Bahrain, Egypt, Jordan, Lebanon, Libya, Saudi Arabia, Syria, Tunisia, UAE, IsraelSources: SART; ESHRE; ICMART; Country Registries; Latin America Registry; Israeli Ministry; Japan Society of Obstetrics and Gynecology
Japan
U.S.
Aus/NZ
Middle East
India, Taiwan, S.K.
Germany
U.K.
France
Italy
Rest of Europe
Turkey
Spain
Lat. AmCanada
Region Cycles
Japan* 326,426
Europe (non-G5) 255,503
United States 174,962
India, Taiwan, S. Korea 148,977
Middle East* 111,788
Germany* 80,943
France 80,349
Australia*/New Zealand 66,347
U.K. 60,473
Italy* 56,147
Turkey* 51,567
Spain 44,896
Latin America 40,710
Canada 25,782
Brazil, Latin America, Russia, Turkey, UAE: Growing 30 - 40% Annually
1.5 Million IVF Cycles in 2013
5
IVFEmbryoFreezing
PGD ICSI IVMEmbryoImaging
IVA
Major Innovations First Discovered and Used Internationally
1981 1992 2011
U.K. Australia U.K. Belgium Denmark Japan
IVF: In Vitro Fertilization
PGD: Pre-implantation Genetic Diagnostics
ICSI: Intracytoplasmic Sperm Injection
IVM: In Vitro Maturation
IVA: In Vitro Activation
1986 1992
Australia
2004
1978 1984 1990 1991 1994 2009 2013 2014
For IVF, Embryo Freezing, PGD, ICSI, IVA and Uterus Transplant: Dates above indicate first baby born
Uterus Transplant
Sweden
6
The EggPCSM Cell Discovery and Opportunity
We always thought a woman was born
with a set number eggs that die over time.
Women have egg precursor (EggPC) cells
in the protective ovarian lining that can
mature into fresh, young, healthy eggs.
This discovery serves as the foundation for
OvaScience’s new fertility treatments.
7
Embryo Cultureand Transfer
Suitability• Blood tests, age, past medical history
Egg Retrieval
Egg Fertilization by ICSI
Ovarian Hyperstimulation• Hormone injections
In Vitro Fertilization (IVF) is the Standard of Care
On average, women go through 2 to 3 IVF cycles
Standard IVF Cycle
ICSI = Intracytoplasmic Sperm Injection
8
IVF Success (% Live Births)
Egg Health and IVF Success Decline with Age
Standard of care (IVF) has 30% success rate
Egg health declines with age, particularly after 35 years
CDC 2009 report on Assisted Reproductive Technology, http://www.cdc.gov/art
0
10
20
30
40
50
60
70
80
22 24 26 28 30 32 34 36 38 40 42 44 46
Age of Women (Years)
Own Eggs
% o
f Li
ve B
irth
s
9
Add energy to eggs
Increase egg reserve
“Next-generation IVF”No hormone injections
AUGMENTSM OvaPrimeSM OvaTureSM
OvaScience Proprietary Fertility Treatments Designed to Improve Egg Health
Launched 2014 End of 2015 Introduction
EggPCCells
In Development
11
Embryo Cultureand Transfer
Suitability• Blood tests, age, past medical history
Egg Retrieval
Egg Fertilization by ICSI
Ovarian Hyperstimulation• Hormone injections
AUGMENTSM Treatment Integrates into IVF Cycle
Standard IVF Cycle
Ovarian tissue biopsy processed
EggPC cells identified and isolated
AUGMENT Process
EggPC mitochondria isolated
+ EggPC Mitochondria
ICSI = Intracytoplasmic Sperm Injection
The AUGMENT treatment is not available in the United States
AUGMENTSM
12
Mitochondria are Key Factor in Egg Health
Younger eggs have younger, healthier mitochondria
Egg health is key factor in IVF success
CDC 2009 report on Assisted Reproductive Technology, http://www.cdc.gov/art
IVF Success (% Live Births)
0
10
20
30
40
50
60
70
80
22 24 26 28 30 32 34 36 38 40 42 44 46
Age of Women (Years)
Own Eggs Donor Eggs
AUGMENTSM
% o
f Li
ve B
irth
s
13
Adding Mitochondria to Egg During IVF Increased IVF Success
1 Huang et al., 1999.2 Cohen et al., 1997, 1998; Brenner et al., 2000; Barritt et al., 2000, 2001.3 Tzeng et al., 2004.4 Levron et al. 5 Lanzendorf et al., 1999.
*Studies conducted in the U.S.
Mitochondria Studies
No. of Cycles Pregnancies Success Rate
Huang1 9 4 44%
Cohen2* 30 13 43%
Tzeng3 71 25 35%
Levron4 15 5 33%
Lanzendorf5* 4 1 25%
AUGMENTSM
Women Who Failed 2 or More IVF Cycles (IVF Success = 0)
14
Previous IVF History AUGMENTSM
Total cycles initiated 71 34
Average cycles initiated per patient 2 1
Total embryo transfers (fresh & frozen) 79 26
Clinical pregnancies 8 12
Clinical pregnancy rate per cycle initiated 11% 35%
Clinical pregnancy rate per embryo transfer 10% 46%
Ongoing clinical pregnancies and live births (rate per cycle initiated)
No ongoing and 1 live birth (1%)
9 including 1 live birth (26%)
AUGMENTSM Clinical Experience Shows Improved Pregnancy Rates Compared to Baseline
Dr. Robert Casper Presentation at COGI Congress, May 2015
# of patients: 34Average current age range: 36 (Range: 26-44)
Presentation includes real-world patient experience from physician’s clinic
The AUGMENT treatment is not available in the United States
AUGMENTSM
9 Patients Have 23 Total Frozen Embryos Remaining for Transfer
15
Additional AUGMENTSM Clinical Experience
Presentation includes real-world patient experience from the physician’s clinic*1 clinical pregnancy ongoing
The AUGMENT treatment is not available in the United States
Physician Stated ≤ 10% Chance of Pregnancy without AUGMENT
Dr. Kutluk Oktay Presentation*
# of patients 8
Patient age range 27-41
# of prior failed IVF cycles 3-7 per patient
Clinical pregnancies/embryo transfers
2/8* = 25%
AUGMENTSM
Dr. Kutluk Oktay Presentation at SRI Meeting, March 2015
16
Foundation for Global Operations Expansion Established
QUALITY
Reliability• Systems control • Compliance• Standardization
across regions• Reproducible• Standard operating
procedures
Flexibility•Design fits clinic
needs• Process development
Scalability• Small footprint•Modular •Modest capital
investment• Fits commercial
strategy to support all fertility treatments
AUGMENTSM
18
Embryo Cultureand Transfer
Suitability• Blood tests, age, past medical history
Egg Retrieval
Egg Fertilization by ICSI
Ovarian Hyperstimulation• Hormone injections
OvaPrimeSM Treatment Process
Standard IVF Cycle
Ovarian tissue biopsy processed
EggPC cells identified and isolated
OvaPrime Process
EggPC cells inserted into ovary
ICSI = Intracytoplasmic Sperm Injection
OvaPrimeSM
19
Major Publications
OvaPrimeSM Treatment: Designed to Increase Egg Reserve
*Includes: Diminished Ovarian Reserve (DOR) of 15-17%; Polycystic Ovarian Syndrome (PCOS) up to
10%; Premature Ovarian Failure/Insufficiency (POF/POI) of 1-3% 1,2,&3
1 SART; ESHRE; ICMART; Country Registries; Latin America Registry; Israeli Ministry2 Am Fam Physician. 2009 Sep 15;80(6):579-5803 In 1986, Carolyn Coulam, M.D. (et al) published an article called Incidence of POF
2011Zhang et al.
2012White et al.
2014 Zhou et al.
2009Zou et al.
OvaPrimeSM
Potential to improve egg health in women with too few or no eggs
– Initial target of 25-30% of all IVF patients*
Treatment Profile
Recent Accomplishments & 2015 Plans
Preclinical proof-of-concept established in vivo
Planning introduction in at least one international region by end of 2015
20
OvaPrimeSM Target Market is Significant
Sources: Human Reprod., CDC Report #73, ESHRE 2012, SART
Patients undergoing treatment
WorldwidePatients
Patients with few or no eggs
Women who produce few eggs frequently do not pursue IVF or do not continue due to low IVF success rates
These patients may consider donor egg or other alternatives
OvaPrime can immediately help by increasing the egg reserve, and hence the response to stimulation
72M
40M
8M
2M
Infertile women
Patients seeking treatment
Initial Target
Market
OvaPrimeSM
21
OvaPrimeSM Treatment Non-Human Primate (NHP) Feasibility
N = 18 NHPs
Stained section of NHP ovary
Preovulatoryfollicle
Primordial/primary follicles
OvaPrimeSM
Isolated EggPC cells using proprietary monoclonal antibody
Optimized EggPC cell delivery into ovaries of autologous NHPs
Resulted in normal physiology following EggPC cell administration
‒ Gross pathology and histology normal at 3 months
Same approach planned to introduce the OvaPrime treatment in patients by end of 2015
22
In Ovary Maturation of Egg Precursor Cells to Mature Eggs in Non-Human Primates (NHPs)
Experiment:
EggPC cells isolated from ovary
EggPC cells labeled with GFP and injected into NHP ovary to mature in vivo
Eggs and tissue harvested after hormone stimulation
Resulted in mature, fertilizable egg
Sources: NIHWolff, et al. Oogonial Stem Cells Generate Mature Oocytes in an Autologous Rhesus Macaque Transplantation Model. Presented at the Meeting of the Society for Gynecologic Investigation; 2014 March 26-29.Wolff, et al. Fertility and Sterility 2013;100(3):S40.
10x
N = 2 NHPs
OvaPrimeSM
24
Embryo Cultureand Transfer
Suitability• Blood tests, age, past medical history
Egg Retrieval
Egg Fertilization by ICSI
Ovarian Hyperstimulation• Hormone injections
OvaTureSM Treatment Process
Standard IVF Cycle
Ovarian tissue biopsy processed
EggPC cells identified and isolated
OvaTure Process
EggPC cells matured in vitro
ICSI = Intracytoplasmic Sperm Injection
OvaTureSM
25
High Throughput Platform To Accelerate OvaTureSM Treatment
Identify and isolate EggPC cells
Evaluate using proprietaryautologous in vitro system
Apply bioinformatics to assess maturation media
Development Platform
OvaTureSM
26
OvaTureSM: Human EggPCSM Cell Maturation to GV Egg
Day 0 Day 1 Day 2 Day 6Day 3
200µm
Supporting cells and green-labeled EggPC cells in vitro
Day 0
GV
Zoom
Note: Every follicle observed showed the same progression
Size and morphology consistent with GV egg
N =45
OvaTureSM
27
Size and morphology consistent with GV egg (preantral follicle)
cell
Initial analysis on day 6 showed follicles present in 100% of wells examined (N>400 follicles)
Additional analysis ongoing
OvaTureSM
Human EggPCSM Cell Matured to GV Egg In Vitro
28
Bovine EggPCSM Cell Matured to GV Egg In Vitro
Size and morphology consistent with GV egg (preantral follicle)
Initial analysis on day 6 showed follicles present in 100% of wells examined (N>300 follicles)
Additional analysis ongoing of different conditions and time points
OvaTureSM
29
Leadership Team
Senior Management Team
Michelle Dipp, M.D., Ph.D. - CEO, Co-FounderSVP, Head CEEDD, GSK VP, Corporate Development, SirtrisM.D., Ph.D., Oxford UniversityBIO Board of Directors and MassBio Advisory Board
Arthur Tzianabos, Ph.D. - PresidentSVP, Research and Non-clinical Development, ShireFaculty, Harvard Medical SchoolPh.D., University of New Hampshire
David Harding - Chief Commercial OfficerGroup SVP and GM of Women’s Health, HologicCytyc Corporation, McKinsey & Company
Theresa McNeely - EVP, Chief Communications OfficerVP, Corporate Communications, Clinical DataMillennium Pharmaceuticals, EXACT Sciences, Pyrosequencing, Allen & Company
Ravi Mehrotra, Ph.D. - Chief Corporate Development OfficerGlobal Head of Biotechnology Equity Research, Credit Suisse SG Cowen, Deutsche Bank (Natwest Markets/BT Alex Brown)Ph.D., Manchester University
Jeffrey Young - Chief Financial OfficerCFO and Treasurer, TransmedicsLantheus Medical Imaging, Critical Therapeutics, PerkinElmer, PricewaterhouseCoopers
Board of Directors
Richard AldrichChairman/Co-FounderLongwood Fund, Biogen, Vertex, Sirtris, Alnara,Verastem, RA Capital
Jeffrey CapelloEVP and CFO of Ortho-Clinical Diagnostics
Michelle Dipp, M.D., Ph.D.
Mary FisherPresident/CEO Colorescience; Skinmedica (sold to Allergan); Acorda, Cephalon, Immunex
Marc KozinFormer President North America, L.E.K. Consulting
Thomas MalleyFounder, Mossrock Capital; Founder, Janus Global Life Sciences Fund
John Sexton, Ph.D.President, New York University
Harald Stock, Ph.D. President and CEO, ArjoHuntleigh; EVP, Getinge Group,; Former CEO Grünenthal Group; DePuy (J&J); Roche
30
EggPC Cells
OvaScience Patent Portfolio
Gene Correction
OvaPrime Treatment
AUGMENT Treatment2 patents extending protection into 2032
OvaTure Treatment
Core Composition of Matter patent into 2025
Method patent for manufacturing into 2025
OvaXon JV
Yale collaboration in endometriosis
Bioenergetic culture media
Additional Patents and Filings Leveraging IP
EggPC Cells